Berotralstat effectiveness and safety in patients with hereditary angioedema with normal C1 inhibitor.

ABSTRACT:

Background

Hereditary angioedema (HAE) with normal C1 inhibitor (nC1-INH) is the least common endotype of HAE, a rare disorder with localized, intermittent attacks of soft tissue swelling. Prophylactic treatments are available across HAE endotypes, including berotralstat, a once-daily oral inhibitor of plasma kallikrein.

Objective

This European multicenter case series aimed to report treatment-related outcomes with berotralstat in patients with HAE-nC1-INH.

Methods

A retrospective case series analysis including observational data from patients in United Kingdom and French centers was performed. Patients were included if they had a genetic assay demonstrating a known HAE-nC1-INH-associated variant; or had a family history of angioedema and normal C1-INH level and funct

SUBMITTER: Buckland MS 

PROVIDER: S-EPMC12573634 | biostudies-literature | 2026 Jan

REPOSITORIES: biostudies-literature